Valoctocogene roxaparvovec-rvox Injection

Valoctocogene roxaparvovec-rvox injection is used to prevent or reduce the frequency of bleeding in certain adults with hemophilia A (condition in which the blood does not clot normally because of a missing or faulty blood clotting factor VIII). Valoctocogene roxaparvovec-rvox is in a class of medications called gene therapy. It works by helping your body to increase blood clotting factor VIII available which may help stop bleeding.

Valoctocogene roxaparvovec-rvox comes as a suspension (liquid) to be injected intravenously (into a vein) by a doctor or nurse in a healthcare setting. It is usually infused slowly over 2 to 5 hours (or longer) as a single dose.

Valoctocogene roxaparvovec-rvox may cause serious reactions during or after you receive your infusion. If you experience any of the following symptoms, tell your doctor immediately: fever, chills, flu-like symptoms, nausea, diarrhea, rash, hives, itching, shortness of breath, difficulty breathing, fast heartbeat, dizziness, feeling faint, or swelling of the face or throat. A doctor or nurse will watch you carefully for these side effects while the medication is being infused, and for at least 3 hours after. Your doctor may need to slow down or temporarily stop your infusion if you experience these or other side effects.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

Before receiving valoctocogene roxaparvovec-rvox injection,

• tell your doctor and pharmacist if you are allergic to valoctocogene roxaparvovec-rvox, mannitol, any other medications, or any of the ingredients in valoctocogene roxaparvovec-rvox injection. Ask your pharmacist for a list of the ingredients.
• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• tell your doctor if you have an infection or have or have ever had hepatitis B or C (a virus that infects the liver and may cause severe liver damage), cirrhosis (scarring) of the liver, or any other liver problems. Your doctor may not want you to receive valoctocogene roxaparvovec-rvox injection.
• tell your doctor if you regularly drink alcohol or sometimes drink large amounts of alcohol, or if you have or ever had blood clots, a stroke, a heart attack or other heart problem, or kidney disease.
• you should know that valoctocogene roxaparvovec-rvox injection is intended only for use in adult men. Valoctocogene roxaparvovec-rvox is not intended for use in women. If you are a male with a female partner who may become pregnant, you should use acceptable birth control during treatment and for 6 months after you receive valoctocogene roxaparvovec-rvox injection. Your doctor will tell you which forms of birth control are acceptable for you to use. Do not donate sperm for 6 months after you receive valoctocogene roxaparvovec-rvox injection. It is not known if valoctocogene roxaparvovec-rvox injection can harm the baby if it is used by women who are pregnant, may become pregnant, or are breastfeeding.
• if you are having surgery, including dental surgery, tell the doctor or dentist that you are receiving valoctocogene roxaparvovec-rvox injection.
• you should not drink alcoholic beverages for at least 1 year after you receive valoctocogene roxaparvovec-rvox injection. Ask your doctor about the safe use of alcoholic beverages 1 year after you receive valoctocogene roxaparvovec-rvox injection. Alcohol can make the side effects from valoctocogene roxaparvovec-rvox worse.
• check with your doctor to see if you need to receive any vaccinations before you receive valoctocogene roxaparvovec-rvox injection. Do not have any vaccinations without talking to your doctor.
• do not donate blood, organs, tissues, or cells for transplantation after you receive valoctocogene roxaparvovec-rvox injection.
• you should know that valoctocogene roxaparvovec-rvox injection may cause liver damage. Your doctor may tell you to have an ultrasound (a type of imaging to look at organs and structures inside the body) of your liver before and after your treatment with valoctocogene roxaparvovec-rvox injection. If you experience any of the following symptoms, call your doctor immediately: extreme tiredness, pain in the upper right part of the stomach, nausea, yellowing of the skin or eyes, dark yellow urine, or pale stools.

Unless your doctor tells you otherwise, continue your normal diet.

Valoctocogene roxaparvovec-rvox injection may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• headache
• stomach pain
• fatigue
• nausea

Some side effects can be serious. If you experience any of these symptoms or those listed in the SPECIAL PRECAUTIONS section, call your doctor immediately or get emergency medical treatment:

• unusual bleeding or bruising
• crushing chest pain
• chest heaviness
• coughing up blood
• shortness of breath
• pain in the back of the lower leg
• feeling of warmth in the lower leg
• sudden changes in vision

Valoctocogene roxaparvovec-rvox injection may increase your risk of developing hepatocellular cancer (a type of liver cancer) or other cancers. Your blood and liver will be monitored at least yearly for at least 5 years after your treatment with valoctocogene roxaparvovec-rvox. Talk to your doctor about the risks of receiving this medication

Valoctocogene roxaparvovec-rvox injection may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to valoctocogene roxaparvovec-rvox injection.

Before having any laboratory test, tell your doctor and the laboratory personnel that you have received valoctocogene roxaparvovec-rvox injection.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.

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